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BioWorld - Monday, December 4, 2023
Breaking News: Topic alerts now available for all BioWorld subscribersBreaking News: ‘Dualing’ obesity drugs: Roche’s $2.7B buyout of Carmot driven by Mounjaro twin mechanismBreaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribers
  • Lung cancer illustration

    RNA-binding plus ribosomal proteins block c-Myc in lung cancer

    Lung adenocarcinoma may be cornered by a recent finding coming from a study published on Nov. 30, 2023, in the Proceedings of the National Academy of Sciences. This investigation has unveiled that a tumor suppressor molecule called RNA-binding motif protein 10 (RBM10) partners with two ribosomal proteins, which in turn inactivate the well-known oncogene Myc proto-oncogene protein (c-Myc).
  • DCZ-5417 suppresses multiple myeloma progression, study shows

  • Cereno completes preclinical safety program for antithrombotic candidate

  • Ebviously presents in vitro data on EBV vaccine candidate

  • Character Biosciences patents new polypeptides for AMD

  • RNA-binding plus ribosomal proteins block c-Myc in lung cancer

    Lung adenocarcinoma may be cornered by a recent finding coming from a study published on Nov. 30, 2023, in the Proceedings of the National Academy of Sciences. This investigation has unveiled that a tumor suppressor molecule called RNA-binding motif protein 10 (RBM10) partners with two ribosomal proteins, which in turn inactivate the well-known oncogene Myc proto-oncogene protein (c-Myc).
  • DCZ-5417 suppresses multiple myeloma progression, study shows

    Norcantharidin is an active ingredient in Chinese traditional medicine that has activity against several cancer types, but its application is limited in the clinic due to toxicity and a narrow treatment window. Researchers from Tongji University and the Shanghai Institute of Materia Medica have recently described a derivative of norcantharidin – DCZ-5417 – for the potential treatment of multiple myeloma (MM).
  • Cereno completes preclinical safety program for antithrombotic candidate

    Cereno Scientific AB has completed the preclinical safety program for CS-014, a histone deacetylase (HDAC) inhibitor in development for arterial and venous thrombosis prevention.
  • Ebviously presents in vitro data on EBV vaccine candidate

    Ebviously GmbH, a spin-off from Helmholtz Munich (HMGU), has presented new in vitro data for its Epstein-Barr virus (EBV) vaccine candidate EBV-001. Based on noninfectious virus-like particles (VLPs) derived from EBV, EBV-001 is designed as a highly immunogenic, multiantigen vaccine to prevent EBV-associated diseases, such as infectious mononucleosis.
  • Character Biosciences patents new polypeptides for AMD

    A Character Biosciences Inc. patent describes polypeptides acting as low-density lipoprotein receptor (LDLR) ligands and reported to be useful for the treatment of age-related macular degeneration (AMD).
  • Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy

    Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and neurologic conditions, including mesial temporal lobe epilepsy (MTLE), among others, leading to an imbalance between excitatory and inhibitory signals.
  • Psychogenics licenses rights to ENT1 inhibitor for neuropsychiatric disorders

    PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential treatment for several psychiatric and neurological conditions.
  • New NLRP3 inflammasome inhibitors reported in Ventus Therapeutics patent

    Ventus Therapeutics US Inc. has disclosed oxoindolinyl amide derivatives acting as NLRP3 inflammasome inhibitors. As such, they are reported to be useful for the treatment of cancer, metabolic, autoimmune diseases, liver, renal, respiratory, cardiovascular and inflammatory disorders, among others.
  • Novel AChE reactivator to reverse acetylcholine accumulation in the brain

    Woman and 3D brain
  • Favorable preclinical profile of investigational gene therapy for TLE reported

  • Wake Forest University designs doxorubicin codrugs with reduced cardiotoxicity

  • TPT-004, a tryptophan hydroxylase inhibitor with efficacy in MC38 mouse colon carcinoma model

    Colorectal cancer illustration
  • Roche divulges EGFR mutant inhibitors for NSCLC

  • Idorsia Pharmaceuticals identifies new orexin OX1 and OX2 receptor antagonists

  • Discovery of dual-target inhibitors of microtubule polymerization and JAK2

    Microscope with laptop displaying histology image.
  • Topic alerts now available for all BioWorld subscribers

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Conferences

  • Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice

    American Association for the Study of Liver Diseases
    LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.
  • Colorized epithelial cells.

    Stem, immune and nervous cells all forces for tumor progression

    Cancer
    One of the difficulties for preventing the evolution of a tumor is that cancer progression can be promoted by undifferentiated or migrating cells whose states could follow different directions. At the 40th edition of Barcelona Biomed conferences at the Institute for Research in Biomedicine (IRB...
  • Neurons and amyloid plaques

    New Alzheimer's disease model includes cholinergic dysfunction

    Neurology/psychiatric
    Researchers from Nagoya City University presented the development of a new mouse model with cholinergic dysfunction and amyloid pathogenesis for Alzheimer's disease.
  • New pemphigus vulgaris mouse model better mimics human disorder

    Dermatologic
    Pemphigus vulgaris is an autoimmune skin disease mediated by autoantibodies to desmoglein-3 (DSG3). Since the adult murine model of this disease is quite limited, researchers aimed to develop a novel active adult pemphigus vulgaris murine model.
More in Conferences

Today's news in brief

  • Respiratory

  • Dermatologic

  • Cardiovascular

  • Hematologic

  • Nephrology

Patents

  • Synrx Therapeutics discovers new POLθ inhibitors for cancer

  • Betta Pharmaceuticals presents new EZH2 inhibitors for cancer

  • Kumquat Biosciences describes new GTPase KRAS inhibitors for cancer

  • Gibson Oncology and Purdue Research Foundation divulge new Topo1 inhibitors and MycG4 ligands

  • Erasca patents new ULK1 inhibitors for cancer

  • Cancer Research Technology discovers new IKK-α inhibitors

  • University of Dundee presents new CDK8 and/or CDK19 inhibitors for cancer

  • Cytokinetics describes new bicyclic piperazinones to modulate skeletal muscle contractility

  • Roche divulges new GABRA2 PAMs for medical imaging of neurological disorders

  • Allianthera (Suzhou) Biopharmaceutical patents new EGFR triple-mutant inhibitors for cancer

Cancer

  • Lung cancer illustration

    China Pharmaceutical University reports discovery of novel EGFR triple-mutation inhibitors

    Most patients with non-small-cell lung cancer (NSCLC) develop acquired drug resistance to EGFR inhibitors. Osimertinib, a third-generation inhibitor, often sees its therapeutic effect reduced due to the C797S mutation of EGFR exon 20, which blocks the binding of Cys797 to osimertinib.
  • Phenomic AI announces collaborations to advance drug discovery for stroma-rich tumors

    Cell therapy
  • New ADC option to treat rare melanoma subtypes divulged

    New compound
  • Dana-Farber Cancer Institute advances allosteric EGFR inhibitor EAI-432

    New compound
  • Anti-GPC1 antibody elicits strong immune response and controls tumor growth in PDAC model

    Antibody
More in Cancer
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Infection

  • Mycobacterium tuberculosis

    Dual Mcl-1 and Bcl-2 inhibition as promising approach for treating tuberculosis

    Tuberculosis still kills a lot of people worldwide (1.6 million deaths per year). Previous findings demonstrated that induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reduced the growth of Mycobacterium tuberculosis in human macrophages, but when inhibiting multiple proteins...
  • New dual inhibitors of host proteases TMPRSS2 and CTSL/CTSB as potential anti-SARS-CoV-2 agents

    Coronavirus
    Current antiviral agents for COVID-19 treatment target viral proteins, which are susceptible to mutation during SARS-CoV-2 evolution. A potential strategy to fight emerging drug resistances is the development of compounds targeting host proteins that are indispensable for the viral life cycle.
  • Ethris gains clearance for phase I study of ETH-47 for respiratory viral infections

    Regulatory
    Ethris GmbH has received approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to proceed with a first-in-human trial of its inhaled mRNA program, ETH-47, in healthy participants for the treatment and prophylaxis of respiratory viral infections.
  • GHRH-R antagonist attenuates pulmonary dysfunction, heart injury in COVID-19 mice

    Conferences
  • Synthesis and evaluation of new quinoline-piperazine/pyrrolidine compounds against leishmaniasis

  • Zenal Pharmachem divulges new compounds to treat infection

    Patents
  • Discovery of TBA derivatives as potent HBV capsid assembly modulators

More in Infection

Neurology/psychiatric

  • Digital spine concept art

    Nurexone reports new siRNA sequences with potential for spinal cord injury

    RNA
    Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
  • Brain penetration profile of Nodthera’s NLRP3 inflammasome inhibitor described

    Inflammatory
    Aberrant NLRP3 inflammasome activation is at the root of a wide number of conditions, from heart, gastrointestinal, kidney or liver disorders to neuroinflammatory diseases such as Parkinson’s disease or multiple sclerosis. Researchers from Nodthera Ltd. have reported on NT-0796, an NLRP3...
  • miR-92a confers protection against inflammation-driven neurodegeneration

    Biomarkers
    MicroRNAs (miRNAs) play an important role in modulating neuronal stress responses, but their impact on neuroprotection has been poorly studied. Multiple sclerosis (MS) is the most common inflammatory disease affecting the central nervous system. Researchers have recently attempted to identify...
  • Shanghai Institute of Organic Chemistry discovers new KEAP1/NRF2/ARE pathway activators

    Patents
    Shanghai Institute of Organic Chemistry has described oleanamide derivatives acting as Kelch-like ECH-associated protein 1 (KEAP1)/nuclear factor erythroid 2-related factor 2 (NFE2-related factor 2; NFE2L2; NRF2)/antioxidant response element (ARE) pathway activators reported to be useful for the...
  • DE Shaw Research describes new TRPA1 antagonists

    Patents
    DE Shaw Research LLC has identified transient receptor potential cation channel subfamily A member 1 (TRPA1) antagonists reported to be useful for the treatment of pain, fibrosis, autoimmune disease, neurological, dermatological, cardiovascular, respiratory and gastrointestinal disorders, among...
More in Neurology/psychiatric

Immune

  • New RIPK2 scaffolding inhibitor selectively blocks microbe-induced inflammation

  • Abbvie reports preclinical profile of kinase inhibitor ABBV-712

  • Aurinia Therapeutics’ AUR-300 proven useful in systemic sclerosis

  • T-Therapeutics raises $59M in series A for T-cell receptors

  • Anti-CD19 KYV-101 shows promise for autoimmune diseases

  • Emergex to support T cell-based Chikungunya vaccine candidate

  • Guangzhou Fermion Technology presents data on TYK2 inhibitor

  • Scirhom submits CTA for anti-iRhom2 antibody for autoimmune disorders

  • TLR7-targeting antibody shows promise in SLE treatment

  • Biocryst outlines preclinical pipeline development

Endocrine/metabolic

  • Alpha-galactosidase enzyme

    FDA clearance for first-in-human study of Uniqure’s AMT-191 for Fabry disease

    Regulatory
    Uniqure NV has received FDA clearance of its IND application for AMT-191, the company’s gene therapy candidate for Fabry disease.
  • CSPC Pharmaceutical’s SYH-2053 receives NMPA clearance for clinical studies in China

    Regulatory
  • Translational data on Regeneron’s leptin receptor agonist support further development

    Monoclonal antibody
  • Study links microbiome, vitamin B12, tissue repair in ulcerative colitis

    Microbiome
  • New AMPK-activating oxazole derivative shows antiadipogenic effects

More in Endocrine/metabolic
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Biomarkers

  • Brain and DNA

    Somatic Ras-MAPK activating variants linked to mesial temporal lobe epilepsy

    Conferences
    Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired...
  • Researchers find de novo CAMK2B variant in patient with tetralogy of Fallot

    Conferences
    CAMK2B encodes calcium/calmodulin-dependent protein kinase type II subunit β, a kinase expressed in the brain that has an important role in synaptic plasticity. Genetic variations are associated with neurodevelopmental disorders.
  • Researchers find KAT6B is deleterious variant behind neuroblastoma

    Conferences
    The GOBACK study analyzed germline susceptibility in children with birth defects and a cancer diagnosis, including a total of 47 probands. Whole-genome sequencing was performed, and researchers evaluated single-nucleotide variants, insertion/deletion variants, as well as copy number variations.
  • HE4 and CA-125 as markers of diffuse myocardial fibrosis

    Conferences
  • POLD3 variant linked to immunodeficiency, neurodevelopmental delay

    Conferences
  • Identification of new candidate causative genes of visual impairment in consanguineous families

    Conferences
  • Mutations in APOA4 gene cause medullary amyloidosis and ADTKD

    Conferences
More in Biomarkers

Gastrointestinal

  • Esperion Therapeutics discovers new ACLY inhibitors

    Patents
    Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer and more.
  • ZHX2 promotes liver recovery after acute liver injury

    Dysfunction in the mitochondria contributes to the development of acute liver injury (ALI). There is emerging evidence indicating the mitochondria is key to maintain liver homeostasis and survival; thus, controlling its functioning is important...
  • Novel hDHODH inhibitor for inflammatory bowel disease described

    Human dihydroorotate dehydrogenase (hDHODH) is a mitochondrial enzyme participating in de novo pyrimidine biosynthesis essential for T- and B-lymphocyte proliferation.
  • Investigators find lactic acid bacteria strain that alleviates IBS in rats

    Irritable bowel syndrome (IBS) is a functional gastrointestinal disorder where visceral hypersensitivity (VH), which causes discomfort and negatively impacts the quality of life, is influenced by stressful events. In recent years, the use of...
  • Lerna Biopharma presents first-in-class GalNAc-siRNA therapeutic for treatment of liver diseases

    Conferences
    Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver...
  • CVI Pharmaceuticals CVI-2742 shows promise for treating NASH

    Conferences
    CVI Pharmaceuticals has presented preclinical data on their thyroid hormone receptor beta (THRB) selective agonist CVI-2742 for the potential treatment of NASH. Selective activation of the THRB contributes to ameliorating the symptoms of...
More in Gastrointestinal

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple backgroundBreakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.


Recent episodes:

  • AI: driving drug development from effective to remarkable
  • The struggle is real: The first half of 2023 was an uphill climb
  • The biosimilars challenge to Humira revs up
  • Preventing opioid overdoses with a smart patch
  • Better times ahead for the biopharma sector? Could be, the new numbers say
  • Radiopharmaceuticals: The next big disrupter?
  • Cambrian carves out a new niche as it works to keep people from getting sick
  • Rethinking obesity: Fitness may be more directly linked to health than weight
  • Looking ahead to 2023: CEOs contemplate the new normal
  • $1B+ biopharma deals keep values afloat, even amid muted volume

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